A new method to replace defective genes in Duchenne muscular dystrophy
Official title: A Novel Technology for Full-Length Gene Replacement Therapy of Duchenne Muscular Dystrophy
Funded at Salk Institute for Biological Studies · NIH-11004357 · Data updated 2026-11-12
This study is testing a new treatment for Duchenne muscular dystrophy (DMD) that uses a harmless virus to deliver a healthy version of a gene to help your muscles work better and stay strong.
Quick facts
| Grant type | Fellowship grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Salk Institute for Biological Studies NIH-funded |
| Lab location | 1 site (La Jolla, UNITED STATES) |
| Project start | February 1, 2022 |
| Project end | January 13, 2026 |
| Funding year | 2025 |
| Award (this year) | $49,544 |
| NIH project number | 5F30HD106732-04 |
| Project ID | NIH-11004357 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are males diagnosed with Duchenne muscular dystrophy, particularly those with specific mutations in the dystrophin gene.
✕Less likely to help
Patients with forms of muscular dystrophy other than Duchenne muscular dystrophy may not benefit from this research.
What this research studies
This research focuses on developing a gene replacement therapy for Duchenne muscular dystrophy (DMD), a severe genetic disorder that leads to muscle degeneration. The approach utilizes Adeno-Associated Viruses (AAV) to deliver a healthy copy of the dystrophin gene directly to muscle cells, aiming to restore their function and prevent further degeneration. By replacing the defective gene rather than editing it, this method seeks to overcome challenges associated with current gene-editing technologies. Patients may be monitored for improvements in muscle function and overall health as part of the therapy.
Why it matters
Potential benefit: If successful, this research could provide a potential cure for Duchenne muscular dystrophy, significantly improving the quality of life and longevity for affected individuals.
How similar studies have gone: Previous research has shown promise in gene replacement therapies for other genetic disorders, indicating potential success for this novel approach in treating DMD.
Where this research is happening
-
Salk Institute for Biological Studies
La Jolla, United States Active
Researchers
- Principal investigator: Hsu, Ryan Hon Hean — Salk Institute for Biological Studies
- Study coordinator: Hsu, Ryan Hon Hean
Related research
Other NIH-funded projects on the same topics.
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Advancing manufacturing and release testing for a Duchenne gene editing therapy
A project to optimize how a dual-vector AAV gene editing treatment for Duchenne muscular dystrophy is made and tested, so it can move toward clinical use.
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Understanding why Duchenne Muscular Dystrophy worsens and how to stop it
A research team at USC is studying Duchenne muscular dystrophy to find out why the disease continues to progress and how to interrupt harmful changes in muscle tissue.
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Stopping steroid-related muscle and bone wasting in Duchenne muscular dystrophy
Researchers are testing ways to protect muscles and bones in people with Duchenne muscular dystrophy who take long-term steroid medicines.
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MR clues to how nNOS affects energy use and blood flow in Becker and Duchenne muscular dystrophy
This project looks at how advanced MRI tests can track muscle energy and blood flow changes in people with Becker and Duchenne muscular dystrophy as new gene therapies are explored.
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Digital motor-skill tracking for infants with Duchenne Muscular Dystrophy using wearables
A wearable-sensor project aims to quietly track and quantify how motor skills develop in babies and young boys with Duchenne Muscular Dystrophy.
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Genetic factors that influence Duchenne Muscular Dystrophy progression
A study to find genes that change how severe Duchenne muscular dystrophy is in boys and young men, using patient data and DNA samples.
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.